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Can a multiple sclerosis drug help people with rare motor neuron disease walk better?
NCT ID NCT02868567
First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This early-stage trial tests the drug dalfampridine (Ampyra), already used for multiple sclerosis, in 35 adults with primary lateral sclerosis or upper motor neuron ALS. The main goal is to see if it safely improves walking speed, measured by a timed 25-foot walk. Researchers will also check effects on quality of life and daily function over 18 weeks.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- dalfampridine (Ampyra)
- What this could lead to
- If it works, this could point toward a treatment to improve walking speed and quality of life for people with primary lateral sclerosis.
- What could go wrong
- This is a very early, small Phase 1 trial with only 35 participants and no placebo group, so results may not be reliable or generalizable. The drug may cause side effects like seizures or allergic reactions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 35 people
The number the study aims to enrol. It can still change while the study runs.
- Start date
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Mar 2016
- Expected to finish
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Jul 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 99 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Male or female, aged 18-99; 2. Diagnosis of upper motor neuron disease, compatible with PLS but may include upper motor neuron (UMN) predominant ALS, defined as only upper motor neuron (UMN) features in at least 2 body regions on examination. 3. EMG within 3 months of enrollment with minimal or no evidence of lower motor neuron disease, 4. Time from symptom onset \> 18 months 5. No previous allergy to dalfampridine 6. No current or exposure to any therapeutic agent targeting PLS or ALS within 30 days of enrollment. 7. Must have a forced vital capacity (FVC) ≥ 60% of expected 8. Written informed consent prior to screening is present. 9. Subjects on a stable dose of or have not taken Riluzole for at least thirty days 10. Impaired walking as measured by a Hauser Index of greater than 1 and less than 7 (2 to 6, inclusive); 11. Mini Mental Status Score \> 22 and deemed by the PI of being capable of providing informed consent and following trial procedures. 12. Geographically accessible to the site. 13. Women must not be able to become pregnant (e.g., post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal contraception, for example patch or contraceptive ring), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method. Exclusion Criteria: 1. History of clinically significant liver disease, renal disease, peripheral neuropathy, serious peripheral vascular disease, known HSP or + C9orf72 or SPG4 mutation, or any other medical condition felt to be exclusionary by the investigator; 2. Unwillingness to sign informed consent or any other reasons for which the investigator feels the subject cannot complete the study; 3. Women who are pregnant, breastfeeding, or trying to become pregnant; 4. Active cancer within the previous 2 years, except treated basal cell carcinoma of the skin; 5. Subjects taking any other experimental drugs within 30 days prior to enrollment; 6. Patient has any history of seizures; brain surgery, brain implants, any metallic implants above the neck, cardiac pacemakers, cochlear implants, piercing or body modification above the neck, known history of TMS related complications or side-effects, tinnitus. 7. Patient has moderate or severe renal impairment as defined by a calculated creatinine clearance of ≤50 mL/minute; 8. Patient has been administered botulinum toxin in the lower extremities within 6 months prior to the screening visit and/or is expected to receive botulinum toxin in the lower extremities during the course of the study; 9. Patient has a known allergy to pyridine-containing substances or any of the inactive ingredients of the dalfampridine tablet (colloidal silicon dioxide, hydroxypropyl methylcellulose, magnesium stearate, microcrystalline cellulose, polyethylene glycol, and titanium dioxide); 10. Patient has a history of drug or alcohol abuse within the past year; 11. Patient has clinically significant abnormal laboratory values. 12. Anything else that, in the opinion of the SI, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Mass General Hospital
Boston, Massachusetts, 02114, United States
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Shara Holzberg
New York, New York, 10021, United States
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University of Florida Gainsville
Gainesville, Florida, 32607, United States